Table 2.
siRNA therapeutic applications in Parkinson’s disease.
| Target gene | Delivery system | Model(s) | Effect(s) | Reference |
|---|---|---|---|---|
|
α-synuclein (SNCA) |
Anionic liposomes decorated with a rabies virus glycoprotein -derived peptide |
In vitro: neuronal cell from P0 newborn C57BL/6J mice |
-Reducing the level of SNCA | [77] |
| Naked siRNA |
In vitro: human neuroblastoma cells (BE(2)-M17) In vivo: wild-type C57BL6 female mice |
-Reducing the level of SNCA | [90]* | |
| Naked siRNA |
In vivo: Primate Substantia Nigra |
Reducing the level of SNCA and the first evidence of successful anti-α-syn uclein intervention in the primate |
[91]* | |
| Viral vector (AAV vectors)) |
In vivo: Thy1-hSNCA mice |
-Decreased hSNCA expression -Rescue of hSNCA-mediated behavioral deficits |
[92]* | |
| ExCont-RNAi |
In vitro: Drosophila S2 cells and human fibroblasts In vivo: flies model of PD |
-Reducing the level of SNCA - Improvement in motor dysfunction |
[93]* | |
| Nanoparticle (LDH) |
In vitro: human neuroblastoma cell line (SH-SY5Y) |
-Reducing the level of SNCA | [165] | |
| PEG-PEI |
In vitro: PC12 cells |
-Protect cells from death via apoptosis | [166] | |
| PEI F25- LMW |
In vitro: human neuroblastoma cell line (SH-SY5Y) In vivo: Thy1-aSyn mice |
-Reducing the level of SNCA | [167] | |
| Peptide mediated delivery |
In vivo:
transgenic mouse model of PD |
-Reducing the accumulation of α-syn -Amelioration of inflammatory pathology |
[168] |
*Explained in the text